{"title":"Public Health Impact and Cost-Effectiveness Analysis of 4-Component Meningococcal Serotype B Vaccination for Infants in France.","authors":"Gérard de Pouvourville, Manon Breau-Brunel, Véronique Loncle-Provot, Ekkehard Beck, Loïg Gaugain, Gaëlle Nachbaur, Céline Pribil","doi":"10.1007/s41669-024-00488-5","DOIUrl":"10.1007/s41669-024-00488-5","url":null,"abstract":"<p><strong>Background and objectives: </strong>In France, meningococcal serogroup B (MenB) is the most common serogroup causing invasive meningococcal disease (IMD) in infants and young children. Our objective was to illustrate the impact of model choices on health outcomes and the cost-effectiveness of infant vaccination with the multicomponent meningococcal serogroup B vaccine (4CMenB) versus no vaccine in France.</p><p><strong>Methods: </strong>A previously published dynamic transmission-based cost-effectiveness model was adapted for the French context using updated, French-specific demographic, epidemiological, and cost data. IMD incidence and long-term sequelae were derived through analysis of French healthcare and surveillance databases. A collective perspective over a 100-year time horizon was adopted, with a discount rate of 2.5%, reduced to 1.5% after the first 30 years. Deterministic and probabilistic sensitivity and scenario analyses were performed.</p><p><strong>Results: </strong>In the base case analysis, infant vaccination with 4CMenB avoided 3101 MenB IMD cases in infants aged < 1 year (- 54%) and 6845 cases in all age groups (- 21%). The estimated incremental cost-effectiveness ratio was €316,272/quality-adjusted life-year (QALY) but was highly sensitive to the types of sequelae included, MenB incidence, vaccine effectiveness parameters, and consideration of life-expectancy in IMD survivors (range: €65,272/QALY to €493,218/QALY).</p><p><strong>Conclusions: </strong>Using economic models compliant with French methodology guidelines, 4CMenB does not seem cost-effective; however, results are sensitive to model choices and 4CMenB immunization is an effective strategy to prevent MenB IMD cases and to improve quality of life and economic burden associated with MenB IMD treatment, especially with regard to long-term sequelae.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252096/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141080591","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-07-01Epub Date: 2024-03-15DOI: 10.1007/s41669-024-00478-7
Sudewi Mukaromah Khoirunnisa, Fithria Dyah Ayu Suryanegara, Lisa Aniek de Jong, Didik Setiawan, Maarten Jacobus Postma
{"title":"A Systematic Review of Health-Related Quality of Life in Women with HER2-Positive Metastatic Breast Cancer Treated with Trastuzumab.","authors":"Sudewi Mukaromah Khoirunnisa, Fithria Dyah Ayu Suryanegara, Lisa Aniek de Jong, Didik Setiawan, Maarten Jacobus Postma","doi":"10.1007/s41669-024-00478-7","DOIUrl":"10.1007/s41669-024-00478-7","url":null,"abstract":"<p><strong>Background: </strong>Many trials of human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer treatment with trastuzumab have provided evidence of improved clinical outcomes. This systematic review examined how a regimen that includes trastuzumab affects patients' health-related quality of life (HRQoL) during and after treatment.</p><p><strong>Methods: </strong>A systematic search for articles published up to February 2023 without restrictions of language or publication year was performed using the Pubmed, Embase, and Scopus databases. We included studies of women aged > 18 years with metastatic HER2-positive breast cancer treated with a trastuzumab-containing regimen. We assessed the quality of the studies using the Cochrane Risk of Bias (RoB) tool (2.0) for randomized controlled trials (RCTs) and the Risk Of Bias In Non-randomised Studies-of Interventions (ROBINS-I) tool for cross-sectional studies. We used Microsoft Excel to extract and synthesize data, and documented the review procedure following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines.</p><p><strong>Results: </strong>In total, eight studies compared 1104 trastuzumab-treated patients and 1003 non-trastuzumab-treated patients. Most studies were RCTs (n = 7) and one was a prospective observational study. All the included studies used the EORTC-QLQ-C30, EORTC-QLQ-BR23, FACT-B, or FACT-G questionnaires. During treatment, patients taking regimens that included trastuzumab showed clinical improvement in HRQoL, social functioning, and role functioning. After the treatment, patients' HRQoL profiles in the trastuzumab and chemotherapy arms were similar. However, trastuzumab (versus chemotherapy) treatment led to clinically improved functional status, role and physical functioning, and fatigue. The quality assessment revealed some concerns in most RCTs, with the risk of bias being high in two studies, low in one study, and moderate in the cross-sectional study.</p><p><strong>Conclusions: </strong>Trastuzumab-containing regimens administered to HER2-positive breast cancer patients at the metastatic stage evidenced beneficial effects on total HRQoL during and after treatment. Upon therapy cessation, patients' HRQoL scores for both interventions improved. Nevertheless, HRQoL profiles of patients treated with trastuzumab were more favorable, particularly for functional status, role functioning, physical well-being, and fatigue.</p><p><strong>Clinical trials registration: </strong>This review was registered in PROSPERO (registration number: CRD42021259826).</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252097/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140137061","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-07-01Epub Date: 2024-03-05DOI: 10.1007/s41669-024-00474-x
Colin Burke, Catriona Crossan, Emma Tyas, Matthew Hemstock, Dawn Lee, Sally Bowditch
{"title":"A Cost-Utility Analysis of Add-On Cannabidiol Versus Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales.","authors":"Colin Burke, Catriona Crossan, Emma Tyas, Matthew Hemstock, Dawn Lee, Sally Bowditch","doi":"10.1007/s41669-024-00474-x","DOIUrl":"10.1007/s41669-024-00474-x","url":null,"abstract":"<p><strong>Objectives: </strong>The aim of this study was to evaluate the cost effectiveness of plant-derived highly purified cannabidiol (Epidyolex<sup>®</sup> in the UK; 100 mg/mL oral solution) as an add-on treatment to usual care for the management of treatment-refractory seizures associated with tuberous sclerosis complex (TSC) in patients aged ≥ 2 years.</p><p><strong>Methods: </strong>A cohort-based model was developed using a National Health Service perspective and lifetime horizon. Health states were based on weekly seizure frequency and seizure-free days, utilizing patient-level data from the GWPCARE6 trial (ClinicalTrials.gov identifier: NCT02544763). Two independent regression models were applied to individual patient-level data to predict seizure-free days and seizure frequency. Healthcare resource utilization data were sourced from a Delphi panel, and patient and caregiver health-related quality of life values were elicited using vignettes valued by the general public. Outcomes relating to TSC-associated neuropsychiatric disorders were modeled with costs and quality-adjusted life-years sourced from published literature.</p><p><strong>Results: </strong>In the base case, compared with usual care alone, 12 mg/kg/day cannabidiol was associated with an incremental cost-effectiveness ratio (ICER) of £23,797. The National Institute for Health and Care Excellence disease severity modifier reduced the ICER to £19,831. Probabilities of cost effectiveness at willingness-to-pay thresholds of £20,000 and £30,000 were 30% and 52%, respectively, for the base case and 39% and 66%, respectively, for the disease severity modifier. Results were robust to sensitivity and scenario analyses.</p><p><strong>Conclusions: </strong>At 12 mg/kg/day and an ICER threshold of £20,000-£30,000, we provide evidence for the cost effectiveness of add-on cannabidiol treatment for patients with TSC-associated seizures aged ≥ 2 years who are refractory to current treatment.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252107/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140028661","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jonathan Broomfield, M Hill, F Chandler, M J Crowther, J Godfrey, M Guglieri, J Hastie, J Larkindale, J Mumby-Croft, E Reuben, F Woodcock, K R Abrams
{"title":"Correction: Developing a Natural History Model for Duchenne Muscular Dystrophy.","authors":"Jonathan Broomfield, M Hill, F Chandler, M J Crowther, J Godfrey, M Guglieri, J Hastie, J Larkindale, J Mumby-Croft, E Reuben, F Woodcock, K R Abrams","doi":"10.1007/s41669-024-00506-6","DOIUrl":"10.1007/s41669-024-00506-6","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252090/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141492950","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-07-01Epub Date: 2024-01-30DOI: 10.1007/s41669-024-00472-z
Rod S Taylor, Anthony Bentley, Kaylie Metcalfe, Melvin D Lobo, Ajay J Kirtane, Michel Azizi, Christopher Clark, Kieran Murphy, Jennifer H Boer, Marjolijn van Keep, An Thu Ta, Neil C Barman, Garrett Schwab, Ron Akehurst, Roland E Schmieder
{"title":"Cost Effectiveness of Endovascular Ultrasound Renal Denervation in Patients with Resistant Hypertension.","authors":"Rod S Taylor, Anthony Bentley, Kaylie Metcalfe, Melvin D Lobo, Ajay J Kirtane, Michel Azizi, Christopher Clark, Kieran Murphy, Jennifer H Boer, Marjolijn van Keep, An Thu Ta, Neil C Barman, Garrett Schwab, Ron Akehurst, Roland E Schmieder","doi":"10.1007/s41669-024-00472-z","DOIUrl":"10.1007/s41669-024-00472-z","url":null,"abstract":"<p><strong>Background: </strong>Resistant hypertension (rHTN) is defined as blood pressure (BP) of ≥ 140/90 mmHg despite treatment with at least three antihypertensive medications, including a diuretic. Endovascular ultrasound renal denervation (uRDN) aims to control BP alongside conventional BP treatment with antihypertensive medication. This analysis assesses the cost effectiveness of the addition of the Paradise uRDN System compared with standard of care alone in patients with rHTN from the perspective of the United Kingdom (UK) health care system.</p><p><strong>Methods: </strong>Using RADIANCE-HTN TRIO trial data, we developed a state-transition model. Baseline risk was calculated using Framingham and Prospective Cardiovascular Münster (PROCAM) risk equations to estimate the long-term cardiovascular risks in patients treated with the Paradise uRDN System, based on the observed systolic BP (SBP) reduction following uRDN. Relative risks sourced from a meta-analysis of randomised controlled trials were then used to project cardiovascular events in patients with baseline SBP ('control' patients); utility and mortality inputs and costs were derived from UK data. Costs and outcomes were discounted at 3.5% per annum. Modelled outcomes were validated against trial meta-analyses and the QRISK3 algorithm and real-world evidence of RDN effectiveness. One-way and probabilistic sensitivity analyses were conducted to assess the uncertainty surrounding the model inputs and sensitivity of the model results to changes in parameter inputs. Results were reported as incremental cost-effectiveness ratios (ICERs).</p><p><strong>Results: </strong>A mean reduction in office SBP of 8.5 mmHg with uRDN resulted in an average improvement in both absolute life-years (LYs) and quality-adjusted life-years (QALYs) gained compared with standard of care alone (0.73 LYs and 0.67 QALYs). The overall base-case ICER with uRDN was estimated at £5600 (€6500) per QALY gained (95% confidence interval £5463-£5739 [€6341-€6661]); modelling demonstrated > 99% probability that the ICER is below the £20,000-£30,000 (€23,214-€34,821) per QALYs gained willingness-to-pay threshold in the UK. Results were consistent across sensitivity analyses and validation checks.</p><p><strong>Conclusions: </strong>Endovascular ultrasound RDN with the Paradise system offers patients with rHTN, clinicians, and healthcare systems a cost-effective treatment option alongside antihypertensive medication.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252101/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139576301","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Public Preference for Financing Preventive Vaccines in Health Shocks: The Case of COVID-19 Vaccines in a Middle-Income Country.","authors":"Najmeh Moradi, Leila Zarei, Narges Hajimoladarvish, Zahra Meshkani, Marzieh Zanganeh, Jafar Babapour, Kamran Bagheri Lankarani","doi":"10.1007/s41669-024-00494-7","DOIUrl":"10.1007/s41669-024-00494-7","url":null,"abstract":"<p><strong>Background: </strong>To effectively manage health crises such as disease pandemics, health authorities require reliable information regarding people's preferences. This helps to ensure timely and targeted interventions and avoids increasing societal costs through developing evidence-based policies. This study investigates the Iranian people's preference for financing coronavirus disease 2019 (COVID-19) vaccines and the factors affecting this.</p><p><strong>Methods: </strong>A cross-sectional survey was performed during the third-wave peak of COVID-19 in Iran from 1 to 20 February 2021 utilizing various data collection methods. The public preference regarding imported and domestically produced vaccines, their willingness to fully or partially contribute to the financing of these vaccines, and their willingness to pay (WTP) for domestically produced vaccines using the contingent valuation method was assessed in different scenarios. The determinants of the probability of positive financing preferences were evaluated using an ordered probit regression model.</p><p><strong>Results: </strong>Among the 2071 survey respondents, approximately 60% stated willingness to contribute to vaccine financing in the form of partial or full user fee payments. Forty percent of respondents are willing to be vaccinated if it is provided for free by the government. Interestingly, people's preference for financing the vaccine was not related to the type of vaccine. The regression analysis showed that income, having supplementary insurance, being male, perceived COVID-19 risk, education, and working in the health sector are significantly related to a higher probability of contribution to vaccine financing.</p><p><strong>Conclusions: </strong>Regardless of country of origin of COVID-19 vaccines, vaccination is very important for the Iranian people, and the majority of respondents in our study showed an inclination to contribute to vaccine funding, from partial to full user fees, to achieve higher protection against COVID-19 disease. Hence, ensuring timely access to vaccines during health crises such as pandemics is imperative, as it saves lives and reduces the economic burden of disease. This commitment from the health system can be supported by financial contributions from the general public. In this regard, considering public preferences is strongly advised to policymakers.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252095/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140958722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Rod S Taylor, Anthony Bentley, Kaylie Metcalfe, Melvin D Lobo, Ajay J Kirtane, Michel Azizi, Christopher Clark, Kieran Murphy, Jennifer H Boer, Marjolijn van Keep, An Thu Ta, Neil C Barman, Garrett Schwab, Ron Akehurst, Roland E Schmieder
{"title":"Correction: Cost Effectiveness of Endovascular Ultrasound Renal Denervation in Patients with Resistant Hypertension.","authors":"Rod S Taylor, Anthony Bentley, Kaylie Metcalfe, Melvin D Lobo, Ajay J Kirtane, Michel Azizi, Christopher Clark, Kieran Murphy, Jennifer H Boer, Marjolijn van Keep, An Thu Ta, Neil C Barman, Garrett Schwab, Ron Akehurst, Roland E Schmieder","doi":"10.1007/s41669-024-00485-8","DOIUrl":"10.1007/s41669-024-00485-8","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252093/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140877018","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-07-01Epub Date: 2024-06-03DOI: 10.1007/s41669-024-00495-6
Carlos Rojas-Roque, Constanza Silvestrini, Fernando Argento, Daniela Sugg, Federico Augustovski, Jesica Coelli, Natalia Espinola
{"title":"Budget Impact Analysis of Olaparib in Combination with Bevacizumab for Maintenance Therapy for Ovarian Cancer in Argentina.","authors":"Carlos Rojas-Roque, Constanza Silvestrini, Fernando Argento, Daniela Sugg, Federico Augustovski, Jesica Coelli, Natalia Espinola","doi":"10.1007/s41669-024-00495-6","DOIUrl":"10.1007/s41669-024-00495-6","url":null,"abstract":"<p><strong>Objectives: </strong>To perform a budget impact analysis (BIA) of introducing olaparib as maintenance therapy in women who have BRCA mutations (BRCAm) with platinum-sensitive recurrent ovarian cancer (PSROC) in combination with bevacizumab in Argentina.</p><p><strong>Methods: </strong>A BIA model was used to analyse over a 5-year time horizon the change in the health system's budget following the adoption of olaparib as maintenance therapy in BRCAm patients with PSROC. The BIA for each year was estimated by comparing the cost difference between the current scenario (treatment with bevacizumab) and the new scenario (the addition of olaparib) for a third-party payer. The BIA is estimated at the national health system level, and by healthcare sectors in Argentina (public sector, social security and private sector). International and national epidemiological data were used to determine the target patient population. Clinical efficacy, safety outcomes and duration of treatments were obtained from the pivotal clinical study report. Relevant direct medical costs were obtained from public data in Argentina and expert consultation. All the costs are reported in US dollars as of October 2022 ($1 = 152.59 Argentine pesos). A scenario analysis assessed the full coverage of the homologous recombination deficiency (HRD) test in Argentina. In addition, one-way sensitivity analysis was conducted to evaluate the model robustness.</p><p><strong>Results: </strong>For a third-party payer with a cohort of 1,000,000 women covered, the estimated target population was 2 individuals in year 1 and 6 individuals in year 5. The incorporation of olaparib, with a wholesale price per pack of $3176.32, was associated with a weighted average of the budget impact per member per month (PMPM) of $0.062 for the national health system, being above the estimated health system budget impact threshold ($0.0153). By healthcare sector, the results of budget impact PMPM for year 5 ranged between $0.08 (public sector) and $0.114 (private sector). For all perspectives, the variables that most influenced the budget impact was the incidence of ovarian cancer, the drug acquisition cost and the treatment duration.</p><p><strong>Conclusions: </strong>The introduction of olaparib for the treatment of BRCAm women with PSROC has a high budget impact for all three health systems in Argentina.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252110/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141237211","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-07-01Epub Date: 2024-05-02DOI: 10.1007/s41669-024-00484-9
Micha J Pilz, Simone Seyringer, Imad Al-Naesan, Madeleine T King, Andrew Bottomley, Richard Norman, Lisa Schlosser, Tobias Hell, Eva Maria Gamper
{"title":"Cancer-Specific Health Utilities: Evaluation of Core Measurement Properties of the EORTC QLU-C10D in Lung Cancer Patients-Data from Four Multicentre LUX-Lung Trials, Applying Six Country Tariffs.","authors":"Micha J Pilz, Simone Seyringer, Imad Al-Naesan, Madeleine T King, Andrew Bottomley, Richard Norman, Lisa Schlosser, Tobias Hell, Eva Maria Gamper","doi":"10.1007/s41669-024-00484-9","DOIUrl":"10.1007/s41669-024-00484-9","url":null,"abstract":"<p><strong>Background: </strong>Cost-utility analysis generally requires valid preference-based measures (PBMs) to assess the utility of patient health. While generic PBMs are widely used, disease-specific PBMs may capture additional aspects of health relevant for certain patient populations. This study investigates the construct and concurrent criterion validity of the cancer-specific European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Utility-Core 10 dimensions (QLU-C10D) in non-small-cell lung cancer patients.</p><p><strong>Methods: </strong>We retrospectively analysed data from four multicentre LUX-Lung trials, all of which had administered the EORTC Quality of Life Questionnaire (QLQ-C30) and the EQ-5D-3L. We applied six country-specific value sets (Australia, Canada, Italy, the Netherlands, Poland, and the United Kingdom) to both instruments. Criterion validity was assessed via correlations between the instruments' utility scores. Correlations of divergent and convergent domains and Bland-Altman plots investigated construct validity. Floor and ceiling effects were assessed.</p><p><strong>Results: </strong>The comparison of the EORTC QLU-C10D and EQ-5D-3L produced homogenous results for five of the six country tariffs. High correlations of utilities (r > 0.7) were found for all country tariffs except for the Netherlands. Moderate to high correlations of converging domain pairs (r from 0.472 to 0.718) were found with few exceptions, such as the Social Functioning-Usual Activities domain pair (max. r = 0.376). For all but the Dutch tariff, the EORTC QLU-C10D produced consistently lower utility values compared to the EQ-5D-3L (x̄ difference from - 0.082 to 0.033). Floor and ceiling effects were consistently lower for the EORTC QLU-C10D (max. 4.67% for utilities).</p><p><strong>Conclusions: </strong>The six country tariffs showed good psychometric properties for the EORTC QLU-C10D in lung cancer patients. Criterion and construct validity was established. The QLU-C10D showed superior measurement precision towards the upper and lower end of the scale compared to the EQ-5D-3L, which is important when cost-utility analysis seeks to measure health change across the severity spectrum.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252099/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140860276","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cost of Care for Non-communicable Diseases: Which Types of Healthcare Providers are the Most Economical in India's Chhattisgarh State?","authors":"Samir Garg, Narayan Tripathi, Kirtti Kumar Bebarta","doi":"10.1007/s41669-024-00489-4","DOIUrl":"10.1007/s41669-024-00489-4","url":null,"abstract":"<p><strong>Background: </strong>Non-communicable diseases (NCDs) affect a large number of people globally and their burden has been growing. Healthcare for NCDs often involves high out-of-pocket expenditure and rising costs of providing services. Financing and providing care for NCDs have become a major challenge for health systems. Despite the high burden of NCDs in India, there is little information available on the costs involved in NCD care.</p><p><strong>Methods: </strong>The study was aimed at finding out the average monthly cost of outpatient care per NCD patient. The average cost was defined as all resources spent directly by government and citizens to get a month of care for a NCD patient. The cost borne by the government on public facilities was taken into account and activity-based costing was used to apportion it to the function of providing outpatient NCD care. For robustness, time-driven activity-based costing and sensitivity analyses were also performed. The study was conducted in Chhattisgarh State and involved a household survey and a facility survey, conducted simultaneously at the end of 2022. The surveys had a sample representative of the state, covering 3500 individuals above age of 30 years and 108 health facilities.</p><p><strong>Results: </strong>The average monthly cost per NCD patient was Indian Rupees (INR) 688 for public providers, INR 1389 for formal for-profit providers and INR 408 for informal private providers and they managed 53.5, 34.3 and 12.0% of NCD patients respectively. The disease profile of patients handled by different types of providers was similar. The average cost per patient was lowest for the primary care facilities in the public sector.</p><p><strong>Conclusions: </strong>The average direct cost of NCD care for government and citizens put together was substantially higher in case of formal for-profit providers compared with public facilities, even after taking into account the government subsidies to public sector. This has implications for allocative efficiency and the desired public-private provider mix in health systems.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252103/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140870788","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}